Correctsequence Therapeutics Co., Ltd

This early study tests a new gene-editing treatment called CS-121 in 15 adults with severe high triglycerides (a type of fat in the blood) that hasn't been controlled by standard treatments. The therapy uses tiny fat particles to deliver a gene editor that targets the APOC3 gene …

This study tests a single dose of CS-101, a gene therapy, in 20 people aged 12-35 with β-thalassemia major, a severe blood disorder requiring regular transfusions. The goal is to see if it is safe and can help patients produce enough healthy red blood cells to stop needing transf…